21 U.S.C. § 360ff–1 — Targeted drugs for rare diseases

(a) Purpose. This section aims to help develop, review, and approve genetically targeted drugs and variant protein targeted drugs. The goal is to meet unmet medical needs for patient subgroups — including groups with different mutations of the same gene — with serious or life-threatening rare diseases. It also aims to make full use of scientific tools like surrogate endpoints and other biomarkers for that purpose. (b) Leveraging of data from previously approved drug application or applications. The FDA may let a sponsor's new application rely on data the same sponsor (or another sponsor who gave them contractual rights to the data) already submitted in an earlier approved application. This only works if the earlier drug used the same or similar genetically targeted technology, or was the same or a similar variant protein targeted drug, as the new one. This must still meet the normal approval standards for drugs or biologics. (c) Definitions. A "genetically targeted drug" is a drug that: is the subject of an application for treating a serious or life-threatening rare disease; may change how a gene or its product works — for example, by suppressing, boosting, or activating it; and uses a genetically targeted technology. "Genetically targeted technology" means non-replicating nucleic acid or similar chemical compounds meant to treat patient subgroups — including those with different mutations in a gene — who share the same disease, even if caused by different variants of that gene. A "variant protein targeted drug" is a drug that: is the subject of an application for treating a serious or life-threatening rare disease; changes how a product of a mutated gene works, where that mutation causes the disease in whole or in part; and is meant to treat patient subgroups, including those with different mutations, who share the same disease. (d) Rule of construction. Nothing in this section changes the FDA's existing authority — including its standards of evidence and conditions for approval — to approve drugs under this chapter or under the Public Health Service Act, as that authority existed before December 13, 2016. It also doesn't create any new right for a sponsor to reference another company's application data beyond what was already allowed before December 13, 2016.